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Life Cycle of Orphan Drug Development & Commercialization

Conducting Effective Orphan Drug Research and Clinical Trials to Expedite Orphan Drug Approval

16-18 Jan 2013
Omni Parker House - Boston, MA, United States of America

Conference Workshop

Two Pre-Conference Workshops on January 16, 2013:

Pre-Conference Workshop A: Integrating Gene Therapy and Stem-Cell Therapy Treatments in Rare Disease Clinical Trials with led by Karen Kozarsky, Ph.D., Vice President, Research and Development at ReGenX Biosciences LLC

Workshop Moderator: Pre-Conference Workshop B: Exploring New Solutions to Reduce the Orphan Drug Funding Gap led by Gary R. Pasternack, M.D., Ph.D., Chief Executive Officer at Askelpion Pharmaceuticals LLC


Why You Should Attend

Life Cycle of Orphan Drug Development & Commercialization

The orphan drug and rare disease industry is one of the hottest topics in modern day medicine. With blockbuster drugs coming off patent, there is a great need for pharmaceutical companies to diversify their portfolios by exploring niche markets. The increasing presence of high-profile manufacturers in the orphan drug industry provides these companies with this opportunity.

The Life Cycle of Orphan Drug Development & Commercialization Conference will focus on the current landscape of rare disease and orphan drug development, different forms of structural based drug designs, and specifically designed clinical trials.Through these clinical trials, and by having connected to the right patients to participate, will increase productivity and expedite orphan drug approval and commercialization of the final product.

By attending this conference, delegates will gain a comprehensive view of the orphan drug and rare disease industry and will have a clearer understanding of the natural histories of rare diseases and how to most effectively treat them. Through maximizing translational research methods to evaluate bio-markers and surrogate markers in the patientís body, this will assist in the design of orphan drug clinical trials resulting in quickly obtaining results and being able to send the orphan drug in for FDA approval.

Attending This Conference Will Enable You To:

1. Evaluate the current rare disease department within the pharmaceutical industry
2. Select the optimal patient target market to engage in rare disease clinical trials
3. Review orphan drug regulations and accelerate orphan drug approval
4. Address the importance of reimbursement and commercialization in the orphan drug industry

Industry leaders attending this conference will benefit from a dynamic presentation format consisting of workshops, panel discussions, and industry-specific case studies that provide accurate, real-world knowledge. Attendees will experience highly interactive conference sessions, 10-15 minutes of Q&A time after each presentation, 4+ hours of networking, and exclusive online access to materials post-event.


Key Topics

  • Improve current rare disease research methods used in early stages of orphan drug development with Genzyme
  • Evaluate where the rare disease research units fit in the portfolio for large and niche pharma companies with Pfizer
  • Utilize translational medicine to be effective in orphan drug development with Vertex
  • Collaborate with patient advocacy groups to increase patient recruitment in clinical trials with BioMarin
  • Review the FDA Safety and Innovation Act and its impact on orphan drug reserach and development with National Organization for Rare Disorders
  • Previous Attendees Include

    marcus evans invites Heads, Vice Presidents, Directors, and Scientists with responsibilities or involvement in the following areas:

    - Rare Disease Discovery
    - Orphan Drug Development
    - Translational Research
    - Research & Development
    - Clinical Operations
    - Regulatory Affairs
    - Reimbursement

    Why Choose marcus evans?

    marcus evans specialises in the research and development of strategic events for senior business executives. From our international network of 63 offices, marcus evans produces over 1000 event days a year on strategic issues in corporate finance, telecommunications, technology, health, transportation, capital markets, human resources and business improvement.

    Above all, marcus evans provides clients with business information and knowledge which enables them to sustain a valuable competitive advantage and makes a positive contribution to their success.

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    Event Partners

    Practical Insights From

    Alvin Shih, M.D., M.B.A.
    Chief Operating Officer, Rare Disease Research Unit

    Seng H. Cheng, Ph.D.
    Head of Research and Early Development, Rare Diseases Science
    Genzyme, a Sanofi Company

    Dennis Dean, Ph.D.
    Senior Vice President and Head Global Exploratory Development
    Vertex Pharmaceuticals

    Serene Josiah Ph.D.
    Director, Discovery Research
    hire Human Genetic Therapies

    C. Frank Bennett Ph.D.
    Senior Vice President, Research
    Isis Pharmaceuticals

    Barbara H. Wuebbels, R.N., M.S.
    Associate Director, Patient Advocacy
    BioMarin Pharmaceutical Inc.

    Kathy Gram, R.N.
    Associate Director, Patient Advocacy
    Millennium: The Takeda Oncology Company

    Diane Dorman
    Vice President, Public Policy
    National Organization for Rare Disorders (NORD)

    Edward M. Kaye, M.D.
    Chief Medical Officer and Senior Vice President
    Sarepta Therapeutics

    Jeffrey T. Walsh
    Chief Operating Officer
    Bluebird Bio Inc.

    Phillip J. Brooks, Ph.D.
    Program Officer, Office of Rare Diseases Research
    National Center for Advancing Translational Sciences (NCATS) National Institutes of Health

    Lyn H. Jones Ph.D., FRSC
    Head of Chemical Biology and Rare Diseases, Chemistry, BioTherapeutics Chemistry

    Nora Yang, Ph.D., MBA
    Director, Program Management and Strategic Operations, Therapeutics for Rare and Neglected Diseases (TRND)
    National Center for Advancing Translational Sciences (NCATS); National Institute of Health

    Susan R. Kahn
    Executive Director
    National Tay-Sachs & Allied Diseases Association (NTSAD)

    Karen Kozarsky, Ph.D.
    Vice President, Research and Development
    ReGenX Biosciences, LLC

    Oved Amitay
    Vice President, Head of Commercial
    Alnylam Pharmaceuticals

    Gary R. Pasternack, M.D., Ph.D.
    Chief Executive Officer
    Asklepion Pharmaceuticals, LLC

    Jamie M. Ring
    Vice President, Patient Advocacy-Rare Diseases
    Genzyme, a Sanofi Company

    Dr. Kiran Meekings
    Consultant, Life Sciences Professional Services
    Thomson Reuters

    Kinnari Patel, PharmD, R.Ph.
    Global Regulatory Sciences, US Regulatory Liaison
    Bristol-Myers Squibb

    ClŠudia Hirawat
    PTC Therapeutics Inc.

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    Event Contact

    For all enquiries regarding speaking, sponsoring and attending this conference contact:

    Robin Yegelwel

    455 N. Cityfront Plaza Dr.
    9th Floor
    Chicago, IL 60611

    (312) 894-6306
    Fax: (312) 894-6304